Genetic Therapy Modes Quiz

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What is the main approach used in gene therapy for diseases caused by recessive mutations?

Complementing the defective gene (correct)

Introducing a normal gene

Targeting the protein product

Silencing the normal gene

What strategy is typically employed for autosomal dominant genetic diseases in gene therapy?

Introducing a normal gene

Activating the mutant protein

Silencing the defective gene (correct)

Increasing expression of the pathogenic gene

Which type of therapeutic RNA inhibits gene function by controlling gene function?

mRNAs

gRNAs

siRNAs (correct)

RNA aptamers

In gene therapy, what are RNAs that reprogram genetic information and include trans-splicing ribozymes used for?

Reprogramming genetic information Signup and view all the answers

What is the main characteristic of siRNAs used in RNA interference?

Double-stranded RNA with a 3′ overhang Signup and view all the answers

Which type of therapeutic RNA is used for DNA modification in gene therapy?

gRNAs Signup and view all the answers

What is the main function of Aptamers in cancer therapy?

Delivering chemotherapeutics to cancer cells Signup and view all the answers

How are SiRNAs generated by the ribonuclease?

Through endonucleolytic processing Signup and view all the answers

What is the main purpose of Ribozymes in gene therapy?

Downregulating pathogenic genes Signup and view all the answers

Which ribozyme motif is characterized by its ability for self-cleavage of a particular phosphodiester bond?

Hammerhead ribozyme Signup and view all the answers

What is the special characteristic of Group I and Group II intron ribozymes?

Self-splicing reaction Signup and view all the answers

Which disease has Ribozymes been clinically trialed for according to the text?

Solid tumors Signup and view all the answers

Genetic Therapy Modes Quiz

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Questions and Answers

What is the main approach used in gene therapy for diseases caused by recessive mutations?

What strategy is typically employed for autosomal dominant genetic diseases in gene therapy?

Which type of therapeutic RNA inhibits gene function by controlling gene function?

In gene therapy, what are RNAs that reprogram genetic information and include trans-splicing ribozymes used for?

What is the main characteristic of siRNAs used in RNA interference?

Which type of therapeutic RNA is used for DNA modification in gene therapy?

What is the main function of Aptamers in cancer therapy?

How are SiRNAs generated by the ribonuclease?

What is the main purpose of Ribozymes in gene therapy?

Which ribozyme motif is characterized by its ability for self-cleavage of a particular phosphodiester bond?

What is the special characteristic of Group I and Group II intron ribozymes?

Which disease has Ribozymes been clinically trialed for according to the text?

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